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皮肤药物开发公司Inflammasome Therapeutics宣布地理萎缩(GA)临床试验的首例患者给药

Inflammasome Therapeutics Announces First Patient Dosed in Geographic Atrophy (GA) Clinical Trial

businesswire | 2024-04-29 | 翻译由动脉网AI生成,点击反馈

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NEWTON, Mass.--(BUSINESS WIRE)--Inflammasome Therapeutics (https://www.inflam.com), a private company developing a new class of inflammasome inhibitor drugs, Kamuvudines, as therapies for prevalent, degenerative diseases, announced the first patient has been dosed in a first-in-class clinical trial for dry AMD.

马萨诸塞州牛顿(BUSINESS WIRE)——炎性体疗法(https://www.inflam.com),一家私营公司开发了一类新的炎性体抑制剂药物卡莫夫定,用于治疗流行的退行性疾病,该公司宣布,第一名患者已在一流的干性AMD临床试验中服用。

The Phase 1 trial (ClinicalTrials.gov ID NCT06164587) is sponsored by the University of Kentucky and expected to enroll up to five patients with GA due to age-related macular degeneration (AMD)..

第一阶段试验(ClinicalTrials.gov ID NCT06164587)由肯塔基大学赞助,预计将招募多达5名因年龄相关性黄斑变性(AMD)而患有GA的患者。。

Trial participants will receive a tiny, sustained release implant (illustration is available) that will provide slow, consistent release of the drug K8 directly into the back of the eye. K8 was specifically designed for retinal delivery and the implants and injector system were crafted to deliver this particular drug.

试验参与者将接受一种微型缓释植入物(可提供插图),该植入物将缓慢,持续地将药物K8直接释放到眼后。K8是专门为视网膜输送而设计的,植入物和注射器系统是专门为输送这种特殊药物而设计的。

This combined drug and delivery strategy allows high therapeutic doses to be maintained in the eye while the drug is undetectable in systemic circulation..

这种联合药物和递送策略允许在体内循环中检测不到药物的同时,在眼睛中维持高治疗剂量。。

“This marks the second trial underway with our Kamuvudines in ophthalmology,” said Dr. Paul Ashton, President and CEO of Inflammasome Therapeutics, developers of both the compounds and delivery systems for administration.

“这标志着卡莫夫定在眼科的第二次试验正在进行中,”炎性体治疗学总裁兼首席执行官保罗·阿什顿博士说,他是该化合物和给药系统的开发人员。

GA affects approximately one million individuals in the US and more than eight million worldwide. In GA, multiple toxic substances (such as complement, amyloid beta, retrotransposons, iron, and reactive oxygen species) build up in the eye and trigger inflammasome activation that then causes cells in the macula to slowly die (atrophy).

GA影响美国约100万人,全球800多万人。在GA中,多种有毒物质(如补体,β淀粉样蛋白,反转录转座子,铁和活性氧)在眼睛中积聚并引发炎性体激活,然后导致黄斑细胞缓慢死亡(萎缩)。

This is a very similar process to what goes on in the brain and nervous system in patients with ALS, Alzheimer’s disease and multiple sclerosis..

这与ALS、阿尔茨海默病和多发性硬化症患者的大脑和神经系统发生的过程非常相似。。

Last year, two drugs (Syfovre and Izervay) were approved for GA that target one of these toxic substances, complement. The drugs do not target other toxic elements and only modestly slow progression of the disease. Unfortunately, they also increase the risk of developing wet AMD. Furthermore, the drugs require an intraocular injection every four to eight weeks..

去年,两种药物(Syfovre和Izervay)被批准用于GA,其靶向这些有毒物质补体之一。这些药物不针对其他有毒元素,只能适度减缓疾病的进展。不幸的是,它们也增加了患湿性AMD的风险。此外,这些药物需要每四至八周进行一次眼内注射。。

There is tremendous interest in developing potential GA treatments. There are 38 other interventional clinical trials for GA registered in clinicaltrials.gov., almost all of which target individual toxic substances, but not the underlying cause of the atrophy, inflammasome activation.

人们对开发潜在的GA治疗产生了极大的兴趣。在clinicaltrials.gov上注册的GA还有38项其他介入性临床试验,几乎所有这些试验都针对个体有毒物质,但不是萎缩的根本原因,即炎性体激活。

“That’s where we believe we provide a distinctive and significant difference,” said Dr. Ashton. “Our Kamuvudines have been shown in pre-clinical studies to block inflammasome activation caused by multiple toxic pathways – complement, amyloid beta, iron overload, retrotransposons, etc. If we can block inflammasome activation in the clinic, we believe we can have a profound effect on the disease by blocking multiple pathways.”.

阿什顿博士说:“这就是我们认为我们提供了一个独特而重要的区别的地方。”。“我们的卡莫夫定在临床前研究中已被证明可以阻断由多种毒性途径引起的炎性体激活-补体,β淀粉样蛋白,铁超负荷,反转录转座子等。如果我们能够在临床上阻断炎性体激活,我们相信我们可以通过阻断多种途径对疾病产生深远的影响。”。

Dr. Ashton confirmed that the implications for treatments in other neuroinflammatory diseases like Alzheimer’s Disease, ALS and Multiple Sclerosis are “extremely interesting. We have Kamuvudines specifically designed for neurological diseases that penetrate into the brain and Central Nervous System from a simple oral tablet.

阿什顿博士证实,对阿尔茨海默氏病、肌萎缩侧索硬化症和多发性硬化症等其他神经炎症疾病的治疗意义“非常有趣。我们有专门为通过简单的口服片剂渗入大脑和中枢神经系统的神经疾病设计的卡莫夫定。

Inflammasome Therapeutics is expected to begin clinical trials in some of these diseases soon as well,” he affirmed..

炎症小体疗法预计也将很快开始对其中一些疾病进行临床试验,”他肯定道。。

Inflammasome Therapeutics’ co-founder, Dr. Jayakrishna Ambati, has spent more than a decade developing Kamuvudines and identifying their role in the inhibition of inflammasome activity that is being found to be the underlying cause of many diseases. In a series of publications in journals such as Science and Nature, he has described the basic research on GA and pre-clinical development of Kamuvudines:.

Inflamasome Therapeutics的联合创始人Jayakrishna Ambati博士花了十多年的时间开发卡穆夫定,并确定了它们在抑制炎症小体活性中的作用,而炎症小体活性是许多疾病的根本原因。在《科学》和《自然》等期刊上发表的一系列出版物中,他描述了GA的基础研究和卡穆夫定的临床前开发:。

Dr. Ambati and Dr. Ashton co-founded Inflammasome Therapeutics in 2016 to develop therapies for prevalent, degenerative diseases and to develop novel delivery technologies for the sustained release of therapeutic agents and compounds. The company combines scientific excellence with proven development expertise and works to develop products via a mixture of licensing agreements and internal development, including work with Boehringer Ingelheim and the Gates Foundation..

Ambati博士和Ashton博士于2016年共同创立了Inflamasome Therapeutics,以开发针对流行性退行性疾病的疗法,并开发用于持续释放治疗剂和化合物的新型递送技术。该公司将卓越的科学知识与成熟的开发专业知识相结合,通过许可协议和内部开发相结合的方式开发产品,包括与勃林格殷格翰和盖茨基金会合作。。

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